Feb 8, 2022 - Pfizer Pipeline

1Breakthroughs that change patients’ l ives

Pfizer Pipeline

Feb 8, 2022

2

Disclaimer

● As some programs are still confidential, some candidates may not be identified in this list. In these materials, Pfizer discloses Mechanism of Action (MOA) information for some candidates in Phase 1 and for all candidates from Phase 2 through regulatory approval. With a view to expanding the transparency of our pipeline, Pfizer is including new indications or enhancements, which target unmet medical need or represent significant commercial opportunities. The information contained on these pages is correct as of Feb 8, 2022.

● Visit www.pfizer.com/pipeline, Pfizer’s online database where you can learn more about our portfolio of new medicines and find out more about our Research and Development efforts around the world.

http://www.pfizer.com/pipeline

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Table of Contents

Pfizer Pipeline Snapshot 4

Inflammation and Immunology 5

Internal Medicine 6-7

Oncology 8-9

Rare Disease 10

Vaccines 11-12

Hospital (Anti-Infectives) 13

Programs Discontinued Since Last Update 14

Backup: Regulatory Designation Definitions 15-16

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Pfizer Pipeline Snapshot

Pipeline represents progress of R&D

programs as of February 8, 2022

▪ 8 programs advanced or are new

▪ 8 programs discontinued since last

update

▪ Included are 59 NMEs, 30 additional

indications

Discovery

Projects

Phase 1

27Phase 2

25Phase 3

27Registration

10Total

89Pfizer Pipeline Snapshot as of

February 8, 2022

Discovery

Projects

Phase 1

27Phase 2

29Phase 3

29Registration

9Total

94Pfizer Pipeline Snapshot as of

November 2, 2021

Recent Approvals

▪ XELJANZ® (tofacitinib) for the treatment of adults w ith active ankylosing spondylitis (AS) (U.S.; E.U.)

▪ Cibinqo® (abrocitinib) for the treatment of moderate-to-severe atopic dermatitis (AD) in adults (U.S; E.U.)

▪ LORVIQUA® (lorlatinib) for the treatment of adult patients w ith ALK- positive advanced NSCLC previously not treated w ith an ALK inhibitor (E.U.)

*- COMIRNA TY® Booster (Pfizer/BioNTech COVID-19 vaccine) expanded Emergency Use Authorization (EUA) from FDA on Dec 9, 2021 to include individuals 16

years of age and older and on Jan 3, 2022 to include individuals 12 years of age and older (U.S.)

- COMIRNATY® received conditional marketing authorization (CMA) from the EMA on Nov 25, 2021 for children aged 5 to 11 years (E.U.)

- PAXLOVID™ (nirmatrelvir [PF-07321332] and ritonavir) received EUA from FDA on Dec 22, 2021 for the treatment of mild-to-moderate COVID-19 in adults and

pediatric patients (12 years of age and older w eighing at least 40 kg [88 lbs]) and w ho are at high risk for progression to severe COVID-19 (U.S.) and CMA from EMA

on Jan 28, 2022 for the treatment of COVID-19 in adults w ho do not require supplemental oxygen and w ho are at increased risk for progressing to severe COVID-19 (E.U.)

Pipeline represents progress of R&D

programs as of November 2, 2021

▪ 13 programs advanced or are new

▪ 16 programs discontinued since last

update

▪ Included are 59 NMEs, 35 additional

indications

Recent Approvals

▪ TICOVAC™ (tick-borne encephalitis (TBE) vaccine) for active immunization to prevent TBE in individuals 1 year of age and older (U.S.)

▪ COMIRNATY® (COVID-19 Vaccine, mRNA) to prevent COVID-19 in individuals 16 years of age and older. COMIRNATY is the f irst COVID-19 vaccine to be granted

approval by the FDA (U.S.)

* COMIRNATY® Booster (Pfizer/BioNTech COVID-19 vaccine) received Emergency Use Authorization (EUA) from FDA on Sep 22, 2021 for individuals 65 years of age

and older, and individuals ages 18 through 64 w ithin certain high-risk groups; received conditional marketing authorization from the EMA on Oct 5, 2021 for 18 years

of age and older.

COMIRNATY® received EUA from FDA (U.S.) for 5 to 11 years old age group on Oct 29, 2021

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Inflammation and Immunology Discovery

Projects

Phase 1

3Phase 2

10Phase 3

1Registration

0Total

14

• Regulatory Designations – See Definitions in Backup

*Clinical trial to be conducted by Philogen S.p.A

►Indicates that the project is either new or has progressed in phase since the previous portfolio update of Pfizer.com

Compound Name Mechanism of Action IndicationPhase of

DevelopmentSubmission Type

ritlecitinib (PF-06651600) JAK3/TEC Inhibitor Alopecia Areata (BREAKTHROUGH) Phase 3 New Molecular Entity

Dekavil* IL-10 Rheumatoid Arthritis (Biologic) Phase 2 New Molecular Entity

PF-06480605 TNFSF15 Blocker Ulcerative Colitis (Biologic) Phase 2 New Molecular Entity

ritlecitinib +/- PF-06650833;

ritlecitinib + tofacitinib

JAK3/TEC Inhibitor

IRAK4 Inhibitor

JAK Inhibitor

Rheumatoid Arthritis Phase 2 New Molecular Entity

PF-06650833 IRAK4 Inhibitor Hidradenitis Suppurativa Phase 2 Product Enhancement

ritlecitinib (PF-06651600) JAK3/TEC Inhibitor Ulcerative Colitis Phase 2 Product Enhancement

ritlecitinib (PF-06651600) JAK3/TEC Inhibitor Crohn's Disease Phase 2 Product Enhancement

ritlecitinib (PF-06651600) JAK3/TEC Inhibitor Vitiligo Phase 2 Product Enhancement

Eucrisa (crisaborole) PDE4 Inhibitor Stasis Dermatitis Phase 2 Product Enhancement

PF-06823859interferon, beta 1, fibroblast

(IFNB1) Blocker

Dermatomyositis (Biologic) (ORPHAN - U.S., E.U.,

PRIME - E.U.)Phase 2 New Molecular Entity

PF-07038124 Topical PDE4 Inhibitor Atopic Dermatitis and Psoriasis Phase 2 New Molecular Entity

PF-06835375 Chemokine Inhibitor Lupus (Biologic) Phase 1 New Molecular Entity

PF-07054894 CCR6 Antagonist Inflammatory Bowel Disease Phase 1 New Molecular Entity

PF-07242813 CD1a inhibitor Atopic Dermatitis (Biologic) Phase 1 New Molecular Entity

6

Internal Medicine (1 of 2)Discovery

Projects

Phase 1

5Phase 2

4Phase 3

1Registration

3Total

13



Myfembree Oral GnRH receptor antagonist Combination with estradiol and norethindrone acetate

for Endometriosis (U.S.)Registration Product Enhancement

►Rimegepant CGRP receptor antagonist Acute migraine (E.U.) Registration New Molecular Entity

►Rimegepant CGRP receptor antagonist Migraine prevention (E.U.) Registration Product Enhancement

Myfembree Oral GnRH receptor antagonist Combination with estradiol and norethindrone acetate

for contraceptive efficacyPhase 3 Product Enhancement

ervogastat (PF-06865571)Diacylglycerol O-Acyltransferase 2 (DGAT2)

Inhibitor

Non-alcoholic Steatohepatitis (NASH) with Liver

Fibrosis Phase 2 New Molecular Entity

ervogastat (PF-06865571) +

clesacostat (PF-05221304)

Diacylglycerol O-Acyltransferase 2 (DGAT2)

Inhibitor; Acetyl CoA-Carboxylase (ACC)

Inhibitor


FibrosisPhase 2 New Molecular Entity

danuglipron (PF-06882961)Glucagon-like peptide 1 receptor (GLP-1R)

AgonistDiabetes Mellitus-Type 2 Phase 2 New Molecular Entity

danuglipron (PF-06882961)Glucagon-like peptide 1 receptor (GLP-1R)

AgonistObesity Phase 2 Product Enhancement



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Internal Medicine (2 of 2)



PF-06946860Growth Differentiation Factor 15 (GDF15)

Monoclonal AntibodyCachexia (Biologic) Phase 1 New Molecular Entity

PF-07081532 Glucagon-like peptide 1 receptor (GLP-1R)

AgonistDiabetes Mellitus-Type 2 and Obesity Phase 1 New Molecular Entity

danuglipron (PF-06882961) +

PF-06865571

Glucagon-like peptide 1 receptor (GLP-1R)

Agonist; Diacylglycerol O-Acyltransferase 2

(DGAT2) Inhibitor



PF-07258669 Melanocortin-4 receptor (MC4R) Antagonist Geriatric Anorexia Phase 1 New Molecular Entity

PF-07202954Diacylglycerol O-Acyltransferase 2 (DGAT2)

Inhibitor





8

Oncology (1 of 2)Discovery

Projects

Phase 1

16Phase 2

4Phase 3

8Registration

0Total

28



Ibrance (palbociclib) CDK 4,6 kinase inhibitor ER+/HER2+ Metastatic Breast Cancer (PATINA) Phase 3Product

Enhancement

sasanlimab (PF-06801591) +

Bacillus Calmette-Guerin (BCG)Anti-PD-1 Non-Muscle-Invasive Bladder Cancer (Biologic) Phase 3 New Molecular Entity

Talzenna (talazoparib) PARP inhibitorCombo w/ Xtandi (enzalutamide) for 1st Line Metastatic

Castration Resistant Prostate CancerPhase 3

Product

Enhancement

Talzenna (talazoparib) PARP inhibitorCombo w/ Xtandi (enzalutamide) for DDR-deficient Metastatic

Castration Sensitive Prostate CancerPhase 3

Product

Enhancement

Xtandi (enzalutamide) Androgen receptor inhibitor Non-metastatic High-Risk Castration Sensitive Prostate Cancer Phase 3Product

Enhancement

Braftovi (encorafinib) +

Erbitux® (cetuximab)

BRAF kinase inhibitor and anti

EGFR1

stline BRAF-mutant Metastatic Colorectal Cancer Phase 3

Product

Enhancement

Braftovi (encorafinib) + Mektovi

(binimetinib) +

Keytruda® (pembrolizumab)

BRAF kinase inhibitor and MEK

inhibitor and anti PD-1

BRAF-mutant Metastatic or Unresectable Locally Advanced

MelanomaPhase 3

Product

Enhancement

Elranatamab BCMA-CD3 Bispecific Antibody Multiple Myeloma Double-Class Exposed (Biologic) Phase 3 New Molecular Entity

Braftovi (encorafinib) + Mektovi

(binimetinib)

BRAF kinase inhibitor and MEK

inhibitor

1st line and 2nd line BRAF-mutant Metastatic Non-Small Cell Lung

CancerPhase 2

Product

Enhancement

ARV-471ER-targeting PROTAC® protein

degraderER+/HER2- Metastatic Breast Cancer Phase 2 New Molecular Entity



• Erbitux® is a registered trademark of ImClone LLC

• Keytruda® is a registered trademark of Merck Sharp & Dohme Corp.

• PROTAC® is a registered U.S. trademark of Arvinas.

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Oncology (2 of 2)



Elranatamab BCMA-CD3 Bispecific Antibody Multiple Myeloma Triple-Class Refractory (Biologic) Phase 2 Product Enhancement

►PF-07901801 (TTI-622) CD47-SIRPα Fusion Protein Hematological malignancies (Biologic) Phase 2 New Molecular Entity

PF-06647020 protein tyrosine kinase 7 (PTK7) Targeted

CytotoxicityCancer (Biologic) Phase 1 New Molecular Entity

PF-06821497 EZH2 Inhibitor Prostate Cancer Phase 1 New Molecular Entity

PF-06873600 CDK 2,4,6 Inhibitor Breast Cancer Metastatic Phase 1 New Molecular Entity

PF-07062119 GUCY2c CD3 Bispecific Antibody Solid Tumors (Biologic) Phase 1 New Molecular Entity

PF-06940434 Integrin alpha-V/beta-8 Antagonist Solid Tumors (Biologic) Phase 1 New Molecular Entity

PF-07209960 interleukin 15 (IL15) Activator Solid Tumors (Biologic) Phase 1 New Molecular Entity

PF-07220060 CDK4 Inhibitor Breast Cancer Metastatic Phase 1 New Molecular Entity

PF-07265807 AXL/MERTK Inhibitor Solid Tumors Phase 1 New Molecular Entity

PF-07104091 CDK2 Inhibitor Breast Cancer Metastatic Phase 1 New Molecular Entity

PF-07248144 KAT6A Epigenetic modifier Breast Cancer Metastatic Phase 1 New Molecular Entity

PF-07284890 BRAF BP kinase Inhibitor Melanoma Phase 1 New Molecular Entity

PF-07284892 SHP2 tyrosine phosphatase Inhibitor Cancer Phase 1 New Molecular Entity

Ibrance + ARV-471CDK 4,6 kinase inhibitor ER-targeting

PROTAC® protein degraderER+/HER2- Metastatic Breast Cancer Phase 1 Product Enhancement

PF-07257876 CD47xPDL1 Bispecific NSCLC (Biologic) Phase 1 New Molecular Entity

►PF-07263689 OBIR-2 Therapeutic Vaccine Solid Tumors (Biologic) Phase 1 New Molecular Entity

►PF-07260437 B7H4-CD3 Bispecific Breast Cancer Metastatic (Biologic) Phase 1 New Molecular Entity


• PROTAC® is a registered U.S. trademark of Arvinas.

10

Rare DiseaseDiscovery

Projects

Phase 1

2Phase 2

2Phase 3

6Registration

1Total

11



somatrogon (PF-06836922) Human Growth Hormone AgonistPediatric Growth Hormone Deficiency (Biologic)

(ORPHAN – U.S., E.U.)Registration New Molecular Entity

PF-07265803p38 Mitogen-Activated Protein Kinase

Antagonist

Dilated Cardiomyopathy due To Lamin A/C Gene

Mutation (ORPHAN - U.S.)Phase 3 New Molecular Entity

fidanacogene elaparvovec

(PF-06838435)Gene Therapy, coagulation factor IX (F9)

Hemophilia (Biologic) (BREAKTHROUGH,

ORPHAN - U.S., E.U., PRIME - E.U.)Phase 3 New Molecular Entity

giroctocogene fitelparvovec

(PF-07055480)Gene Therapy, coagulation factor VIII (F8)

Hemophilia (Biologic) (RMAT, FAST TRACK,

ORPHAN - U.S., E.U.)Phase 3 New Molecular Entity

somatrogon (PF-06836922) Human Growth Hormone AgonistAdult Growth Hormone Deficiency (Biologic)

(ORPHAN - U.S., E.U.)Phase 3 Product Enhancement

fordadistrogene movaparvovec

(PF-06939926)Gene Therapy, minidystrophin

Duchenne Muscular Dystrophy Ambulatory

(Biologic) (FAST TRACK – U.S.; ORPHAN - U.S.,

E.U.)

Phase 3 New Molecular Entity

marstacimab (PF-06741086) Tissue Factor Pathway Inhibitor (TFPI)Hemophilia (Biologic) (FAST TRACK –

U.S.; ORPHAN - U.S., E.U.)Phase 3 New Molecular Entity

PF-06730512fusion protein containing SLIT ligand

portion of ROBO2 receptor

Focal Segmental Glomerulosclerosis (FSGS);

ROBO2-Fc (Biologic)Phase 2 New Molecular Entity

reciferceptSoluble recombinant human fibroblast

growth factor receptor 3 (FGFR3) decoyAchondroplasia (Biologic) (ORPHAN - U.S., EU) Phase 2 New Molecular Entity

PF-06755347 Immunomodulation

Chronic Inflammatory Demyelinating

Polyneuropathy (ORPHAN-US); Primary Immune

Thrombocytopenia (Biologic)

Phase 1 New Molecular Entity

PF-07209326 E-Selectin antagonist Sickle Cell Disease (Biologic) (ORPHAN - U.S.) Phase 1 New Molecular Entity



11

Vaccines (1 of 2)Discovery

Projects

Phase 1

1Phase 2

4Phase 3

8Registration

5Total

18

1. Comirnaty (Pfizer/BioNTech COVID-19 vaccine) received Emergency Use Authorization (EUA) from FDA on Dec 11, 2020 and FDA BLA on Aug 23, 2021 for 16 years of age and older; received conditional marketing authorization (CMA) from the

EMA on Dec 21, 2020 for 16 years of age and older. 12-15 years old age group received EUA from FDA on May 10, 2021 and conditional marketing authorization from the EMA on May 28, 2021.

2. COMIRNATY® Booster (Pfizer/BioNTech COVID-19 vaccine) expanded Emergency Use Authorization (EUA) from FDA on Dec 9, 2021 to incl ude individuals 16 years of age and older and on Jan 3, 2022 to include individuals 12 years of age and

older; received CMA from the EMA on Oct 5, 2021 for 18 years of age and older.

3. Comirnaty (Pfizer/BioNTech COVID-19 vaccine) received EUA from FDA on Oct 29, 2021 and CMA from EMA on Nov 25, 2021 for 5 to 11 years of age

4. In February 2022, following a request from the FDA, a roll ing submission seeking to amend the Comirnaty EUA to include children 6 months through 4 years of age (6 months to <5 years of age) was initiated. This application is for authorization of the

first two 3 µg doses of a planned three-dose primary series in this age group. Data on a third dose given at least 8 weeks after completion of the second dose are expected in the coming months and will be submitted to the FDA to support a potential

expansion of this requested EUA





Comirnaty (Covid-19 Vx) Prophylactic mRNA Vaccine

COVID-19 Infection (in collaboration with BioNTech) (FAST

TRACK, U.S. – 12 to 15 years of age) (E.U. – 12 years of age

and older)1Registration Product Enhancement

Comirnaty (Covid-19 Vx) Prophylactic mRNA VaccineCOVID-19 Infection Booster (in collaboration with

BioNTech) (FAST TRACK, U.S.; EU)2 Registration Product Enhancement

Comirnaty (Covid-19 Vx) Prophylactic mRNA VaccineCOVID-19 Infection (in collaboration with BioNTech) (FAST

TRACK, U.S.; EU – 5 to 11 years of age)3 Registration Product Enhancement

►Comirnaty (Covid-19 Vx) Prophylactic mRNA VaccineCOVID-19 Infection (in collaboration with BioNTech) (FAST

TRACK, U.S – 6 months to 4 years of age)4 Registration Product Enhancement

PF-06482077 Prophylactic VaccineInvasive and Non-Invasive Pneumococcal infections (adult)

(E.U.)Registration New Molecular Entity

PF-06425090 Prophylactic Vaccine Primary Clostridioides difficile infection (FAST TRACK) Phase 3 New Molecular Entity

PF-06482077 Prophylactic VaccineInvasive and Non-Invasive Pneumococcal infections

(pediatric) (BREAKTHROUGH, FAST TRACK)Phase 3 Product Enhancement

PF-06928316 Prophylactic VaccineRespiratory Syncytial Virus Infection (maternal) (FAST

TRACK)Phase 3 New Molecular Entity

PF-06886992 Prophylactic VaccineSerogroups ABCWY Meningococcal Infections (adolescent

and young adults)Phase 3 New Molecular Entity

PF-06928316 Prophylactic Vaccine Respiratory Syncytial Virus Infection (older adult) Phase 3 Product Enhancement

12

Vaccines (2 of 2)





Comirnaty (Covid-19 Vx) Prophylactic mRNA VaccineCOVID-19 Infection (in collaboration with BioNTech) (children

2 to 4 years of age)Phase 3 Product Enhancement

Comirnaty (Covid-19 Vx) Prophylactic mRNA VaccineCOVID-19 Infection (in collaboration with BioNTech) (infants 6

months to <24 months)Phase 3 Product Enhancement

►Omicron variant (Covid-19

Vx)Prophylactic mRNA Vaccine COVID-19 Infection (in collaboration with BioNTech) (adults) Phase 3 New Molecular Entity

PF-06842433 Prophylactic VaccineInvasive and Non-Invasive Pneumococcal infections (infants

and children)Phase 2 New Molecular Entity

PF-06760805 Prophylactic VaccineInvasive Group B Streptococcus Infection (maternal) (FAST

TRACK)Phase 2 New Molecular Entity

PF-07307405 Prophylactic Vaccine Lyme disease (FAST TRACK) Phase 2 New Molecular Entity

PF-06886992 Prophylactic Vaccine Serogroups ABCWY Meningococcal Infections (infants) Phase 2 Product Enhancement

PF-07252220 Prophylactic mRNA Vaccine Influenza (adults) Phase 1 New Molecular Entity

13

Hospital (Anti-Infectives)Discovery

Projects

Phase 1

0Phase 2

1Phase 3

3Registration

1Total

5



►PaxlovidSARS-CoV-2 3CL protease inhibitor

(oral COVID-19 treatment)

COVID-19 Infection (high risk population) (FAST

TRACK, U.S.; EU)*Registration New Molecular Entity

aztreonam-avibactam (PF-06947387) Beta Lactam/Beta Lactamase InhibitorTreatment of infections caused by Gram-negative

bacteriaPhase 3 New Molecular Entity

PaxlovidSARS-CoV-2 3CL protease inhibitor

(oral COVID-19 treatment)COVID-19 Infection (standard risk population) Phase 3 Product Enhancement

PaxlovidSARS-CoV-2 3CL protease inhibitor

(oral COVID-19 treatment)COVID-19 Infection (post exposure prophylaxis) Phase 3 Product Enhancement

Fosmanogepix (APX001) Gwt1 inhibitor Treatment of invasive fungal infections Phase 2 New Molecular Entity

*PAXLOVID™ (nirmatrelvir [PF-07321332] and ritonavir) received EUA from FDA on Dec 22, 2021 for the treatment of mild-to-moderate COVID-19 in adults and pediatric patients (12 years of age and older weighing at least 40

kg [88 lbs]) and who are at high risk for progression to severe COVID-19 (U.S.) and CMA from EMA on Jan 28, 2022 for the treatment of COVID-19 in adults who do not require supplemental oxygen and who are at increased

risk for progressing to severe COVID-19


14

Programs Discontinued from Development since November 2, 2021



Bavencio (avelumab) Anti PD-L1 1st

Line Non-Small Cell Lung Cancer (Biologic) Phase 3 Product Enhancement

Bavencio (avelumab) Anti PD-L1

Combo w/Talzenna (talazoparib) for Locally

Advanced (Primary or Recurrent) or Metastatic

Solid Tumors (Biologic)

Phase 2 Product Enhancement

Bavencio (avelumab) Anti PD-L1Combo w/Talzenna (talazoparib) for Solid Tumors

with a BRCA or ATM defect (Biologic)Phase 2 Product Enhancement

Talzenna (talazoparib) PARP inhibitor2

ndLine Metastatic Castration-Resistant Prostate

CancerPhase 2 Product Enhancement

vupanorsen (PF-07285557)Angiopoietin Like 3 (ANGPTL3)

Antisense Oligonucleotide

Severe Hypertriglyceridemia, Cardiovascular Risk

ReductionPhase 2 New Molecular Entity

PF-07304814SARS-CoV-2 3CL protease inhibitor (IV

COVID-19 treatment)COVID-19 Infection Phase 2 New Molecular Entity

PF-07059013 Hemoglobin, Beta (HBB) Modulator Sickle Cell Disease (ORPHAN - U.S.) Phase 1 New Molecular Entity

PF-06939999protein arginine methyltransferase 5

(PRMT5) InhibitorSolid Tumors Phase 1 New Molecular Entity

*In the fourth quarter of 2021, enrollment was stopped in C4591015 Study (a Phase 2/3 placebo controlled randomized observer-blind study to evaluate the safety, tolerability, and immunogenicity of BNT162b2 against COVID-19 in healthy pregnant women

18 years of age and older). This study was developed prior to availability or recommendation for COVID-19 vaccination in pregnant women. The environment changed during 2021 and by September 2021, COVID-19 vaccines were recommended by

applicable recommending bodies (e.g., ACIP in the U.S.) for pregnant women in all participating/planned countries, and as a result the enrollment rate declined significantly. With the declining enrollment, the study had insufficient sample size to assess the

primary immunogenicity objective and continuation of this placebo controlled study could no longer be justified due to global recommendations. This proposal was shared with and agreed to by FDA and EMA.

15Breakthroughs that change patients’ l ives

Back-up

16

Regulatory Designations● Fast Track (U.S.) is a designation available to a product if it is intended, whether alone or in combination with one or more other drugs, for the treatment of a serious or life-threatening disease or

condition, and it demonstrates the potential to address unmet medical needs for such a disease or condition. This designation is intended to facilitate development and expedite review of drugs to treat serious and life-threatening conditions so that an approved product can reach the market expeditiously. More information about the qualifying criteria and features of the Fast Track program can be found on the FDA’s website.

● Breakthrough Designation (U.S.) may be granted to a drug (alone or in combination with 1 or more other drugs) intended to treat a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development. A drug that receives breakthrough designation is eligible for all fast track designat ion features and an FDA commitment to work closely with the sponsor to ensure an efficient drug development program. More information about the qualifying criteria and features of the Breakthrough program can be found on the FDA’s website.

● Orphan Drug (U.S.) - Orphan drug status may be granted to drugs and biologics that are intended for the diagnosis, prevention, or treatment of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but where it is unlikely that expected sales of the product would cover the sponsor’s investment in its development. More information about the qualifying criteria, features, and incentives involved in an orphan drug designation can be found on the FDA’s website.

● Orphan Drug (E.U.) - Orphan drug status may be granted to drugs and biologics that are intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition affecting no more than 5 in 10,000 persons in the European Union at the time of submission of the designation application, or that affect more than 5 in 10,000 persons but where it is unlikely that expected sales of the product would cover the investment in its development. More information about the qualifying criteria, features, and incentives involved in an orphan drug designation can be found on the EMA’s website.

● A U.S. drug application will receive a priority review designation if it is for a drug that treats a serious condition and, if approved, would provide a significant improvement in safety or effectiveness. A priority designation is intended to direct overall attention and resources to the evaluation of such applications. A priorit y review designation means that FDA’s goal is to take action on the marketing application within 6 months of receipt (compared with 10 months under standard review). More information about the qualifying criteria and features of a priority review designation can be found on the FDA’s website.

● PRIME (E.U.) - The PRIME scheme is applicable to products under development which are innovative and yet to be placed on the EU market. The scheme aims to support medicinal products of major public health interest and in particular from the viewpoint of therapeutic innovation. Medicines eligible for PRIME must address an unmet medical need, i.e. for which there exists no satisfactory method of diagnosis, prevention or treatment in the Community or, if such a method exists, in relation to which the medicinal product concerned will be of major therapeutic advantage to those affected. A product eligible for PRIME should demonstrate the potential to address, to a significant extent, the unmet medical need, for example by introducing new methods of therapy or improving existing ones. Data available to support the request for eligibility should support the claim to address the unmet medical need through a clinically meaningful improvement of efficacy, such as having an impact on the prevention, onset or duration of the condition, or improving the morbidity or mortality of the disease. EMA will provide early and enhanced support to optimize the development of eligible medicines. Products granted PRIME support are anticipated to benefit from the Accelerated Assessment procedure. More information about the qualifying criteria and features of PRIME and Accelerated Assessment can be found on the EMA’s website.

● Regenerative Medicine Advanced Therapy (RMAT) (U.S.) is a designation that is granted to regenerative medicine therapies intended to treat, modify, reverse, or cure a serious condition, for which preliminary clinical evidence indicates that the medicine has the potential to address an unmet medical need. The RMAT designation includes all the benefits of the fast track and breakthrough therapy designation programs, including early interactions with FDA.

Documents

Feb 8, 2022 - Pfizer Pipeline